Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
|ClinicalTrials.gov Identifier: NCT00885833|
Recruitment Status : Completed
First Posted : April 22, 2009
Last Update Posted : July 14, 2014
|Condition or disease||Intervention/treatment||Phase|
|Wiskott-Aldrich Syndrome||Drug: Fludarabine, Busulfan, Thymoglobulin||Phase 1 Phase 2|
Wiskott-Aldrich syndrome (WAS) is an rare X-linked congenital immune-deficiency syndrome characterized by the triad of recurrent infection, eczema and thrombocytopenia with small size of platelet (Puck JM, 2006). Clinical studies revealed high rate of autoimmune disorder and malignancy in WAS (Ochs HD, 2006). The identification of the molecular defect in 1994 (Derry JM, 1994) has broadened the clinical spectrum of the syndrome to include chronic or intermittent X-linked thrombocytopenia (XLT), a relatively mild form of WAS and X-lined neutropenia caused by an arrest of myelopoiesis (Ochs HD, 2006).
The incidence of WAS in Korea was very low and only 6 patients diagnosed between 2001 and 2005 (Kim JG, 2006).
Conventional treatments for WAS such as prophylactic antibiotics and immune globin for infection and platelet transfusion for bleeding were not so successful (Thrasher AJ, 2000). Bone marrow transplantation (BMT) from an HLA-matched related donor is an effective treatment (Filipovich AH, 2001) and patients without appropriate related donor could receive alternative stem cell source such as matched unrelated donor or cord blood. But the transplant with the alternative donor needed more intensive conditioning to overcome the hematologic and immunologic barrier with increased treatment related toxicity. Further progress depends in particular on the development of alternative preparative conditioning regimens which allow stable engraftment of donor precursor cells with minimal systemic toxic side effects (Friedrich W, 2004).
Recently, we reported successful unrelated bone marrow transplantation in a boy with WAS with reduced toxicity myeloablative conditioning regimen to increase the engraftment potential without serious complication (Kang, 2008), and extended to multicenter phase I/II pilot study with this reduced toxicity myeloablative conditioning regimen in the HSCT for WAS.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome|
|Study Start Date :||February 2007|
|Actual Primary Completion Date :||March 2012|
|Actual Study Completion Date :||March 2012|
Drug: Fludarabine, Busulfan, Thymoglobulin
fludarabine (40 mg/m2 once daily i.v. on days -8, -7, -6, -5, -4 & -3) busulfan (0.8 mg/kg every 6 hours i.v. on days -6, -5, -4, & -3) thymoglobulin (2.5 mg/kg once daily i.v. on days -8, -7, -6 for cord blood and on days -4, -3, -2 for bone marrow or mobilized peripheral blood)
- To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for HSCT in WAS. [ Time Frame: Feb. 2007 to Jan. 2012 ]
- To evaluate the incidence and severity of toxicity and treatment related mortality. [ Time Frame: Feb. 2007 to Jan. 2012 ]
- To evaluate overall and event free survival rate. [ Time Frame: Feb. 2007 to Jan. 2012 ]
- To evaluate acute and chronic graft versus host disease (GVHD). [ Time Frame: Feb. 2007 to Jan. 2012 ]
- To evaluate immunologic recovery after HSCT. [ Time Frame: Feb. 2007 to Jan. 2012 ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00885833
|Korea, Republic of|
|Seoul National University Hospital|
|Seoul, Chongno-gu, Korea, Republic of, 110-744|
|Principal Investigator:||Hyo Seop Ahn, Ph. D||The Korean Society of Pediatric Hematology Oncology|